A US Phase 3b, Multi-center, Randomized, Double-blind, Double-Dummy Study to Evaluate Efficacy of Remibrutinib Compared to Dupilumab at Early Timepoints in Adults With Chronic Spontaneous Urticaria Inadequately Controlled by Second Generation H1-Antihistamines

This is a US, multi-center, randomized, double-blind, double-dummy, Phase 3b study to evaluate efficacy of remibrutinib (25 mg twice daily [b.i.d.] by mouth [p.o.]) compared to dupilumab (600 mg loading dose administered subcutaneously (s.c.) followed by 300 mg every 2 weeks s.c.) at early timepoints (4 weeks and earlier), when administered as an add-on treatment to second generation H1-antihistamines (sgH1-AH) (standard label dose as background therapy) in adult US participants with moderate to severe chronic spontaneous urticaria (CSU) inadequately controlled by sgH1-AHs.

Start Date25 Jul 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT06846281

/ Not yet recruitingPhase 3

A Randomized, Open-label, Parallel-group, Non-inferiority Study Comparing Efficacy, Safety, and Tolerability of Remibrutinib After Switching From Ocrelizumab in Participants Living With Relapsing Multiple Sclerosis, Followed by Open-label Treatment With Remibrutinib

The purpose of this Phase 3b study is to assess the efficacy, safety and tolerability of remibrutinib after switching from ocrelizumab and compared to continuous ocrelizumab treatment, in patients living with relapsing multiple sclerosis (plwRMS).

Start Date16 Jun 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT06865651

/ Not yet recruitingPhase 2

A 12-week Randomized, Participant and Investigator-blinded, Placebo-controlled, Exploratory Study in Adult Participants With Chronic Urticaria to Assess the Efficacy and Safety and Explore the Mechanism of Action of Remibrutinib (LOU064)

The purpose of this study is to explore the effect and Mechanism of Action (MoA) of remibrutinib (LOU064) vs. placebo on clinical outcomes in participants with Chronic Urticaria (CU), including both Chronic Spontaneous Urticaria (CSU) and Chronic Inducible Urticaria (CINDU).

Start Date03 Jun 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

100 Clinical Results associated with Remibrutinib

100 Translational Medicine associated with Remibrutinib

100 Patents (Medical) associated with Remibrutinib

Literatures (Medical) associated with Remibrutinib

06 May 2025·JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Remibrutinib Shows Promise for Chronic Hives Relief

Article

Author: Anderer, Samantha

01 May 2025·Clinical and Translational Allergy

Biological and target synthetic treatments for chronic spontaneous urticaria: A systematic review and network meta‐analysis

Article

Author: Song, Xiaoting ; Peng, Chengyue ; Zhang, Peixin ; Zheng, Yaqi ; Zhang, Litao ; Huang, Xiaojie ; Tan, Yen ; Zhao, Zuotao ; Liao, Shuanglu

Abstract:

Background:

Most biological and synthetic target‐specific drugs for antihistamine‐refractory chronic spontaneous urticaria (CSU) have not been compared head‐to‐head. This systematic review and network meta‐analysis evaluated their relative efficacy and safety.

Methods:

Searches were conducted on PubMed, Embase, Web of Science and Cochrane library databases to March 25, 2024 for randomized trials. A random‐effects model was used to calculate the network estimates reported as mean differences (MD) and odds ratios (OR) with corresponding 95% CIs. Main outcomes included the weekly urticaria activity score (UAS7), adverse events (AEs) and serious adverse events (SAEs).

Results:

23 randomized clinical trials with 6933 participants that compared 26 different interventions or dosages and placebos were included. Omalizumab 300 mg [MD −10.07, 95% CI (−11.35; −8.82)] continues to demonstrate superior efficacy compared with placebo. Remibrutinib, at doses of 35 mg once daily [MD −7.80, 95% CI (−12.76; −2.51)], 25 mg twice daily [MD −7.69, 95% CI (−9.85; −5.76)], and 10 mg twice daily [MD −7.61, 95% CI (−12.59; −2.47)], had the best overall performance for efficacy and safety. Dupilumab, fenebrutinib, and rilzabrutinib also showed greater efficacy than placebo. The results were similar for the proportion of patients who achieved UAS7 ≤ 6 or UAS7 = 0. No significant differences were found among all treatment comparisons for AEs and SAEs.

Conclusion:

The findings of this study indicate that the biological agent omalizumab 300 mg and the oral small molecule remibrutinib at doses of 35 mg, 25 mg, or 10 mg are recommended for patients with antihistamine‐refractory CSU.

Trial Registration:

PROSPERO: CRD42024516289

11 Apr 2025·ACS Pharmacology & Translational Science

Comprehensive Characterization of Bruton’s Tyrosine Kinase Inhibitor Specificity, Potency, and Biological Effects: Insights into Covalent and Noncovalent Mechanistic Signatures

Review

Author: Servant, Nicole B. ; King, Alastair J. ; Jahic, Mirza ; Darragh, Antonia C. ; Lipner, Justin H. ; Hanna, Andrew M.

Uncovering a drug's mechanism of action and possible adverse effects are critical components in drug discovery and development. Moreover, it provides evidence for why some drugs prove more effective than others and how to design better drugs altogether. Here, we demonstrate the utility of a high-throughput in vitro screening platform along with a comprehensive panel to aid in the characterization of 15 Bruton's tyrosine kinase (BTK) inhibitors that are either approved by the FDA or presently under clinical evaluation. To compare the potency of these drugs, we measured the binding affinity of each to wild-type BTK as well as a clinically relevant resistance mutant of BTK (BTK C481S). In doing so, we discovered a considerable difference in the selectivity and potency of these BTK inhibitors to the wild-type and mutant proteins. Some of this potentially contributes to the adverse effects experienced by patients undergoing therapy using these drugs. Overall, noncovalent BTK inhibitors showed stronger potency for both the wild-type and mutant BTK when compared with that of covalent inhibitors, with the majority demonstrating a higher specificity and less off-target modulation. Additionally, we compared biological outcomes for four of these inhibitors in human cell-based models. As expected, we found different phenotypic profiles for each inhibitor. However, the two noncovalent inhibitors had fewer off-target biological effects when compared with the two covalent inhibitors. This and similar in-depth preclinical characterization of drug candidates can provide critical insights into the efficacy and mechanism of action of a compound that may affect its safety in a clinical setting.

News (Medical) associated with Remibrutinib

29 Apr 2025

·www.globenewswire.com

Novartis continues strong momentum with double-digit sales growth, robust margin expansion and multiple approvals in Q1

Ad hoc announcement pursuant to Art. 53 LR Net sales grew +15% (cc1, +12% USD) with core operating income1 up +27% (cc, +23% USD) Sales growth driven by continued strong performance from Entresto (+22% cc), Kisqali (+56% cc), Kesimpta (+43% cc), Cosentyx (+18% cc), Leqvio (+72% cc) and Scemblix (+76% cc) Core operating income margin1 reached 42.1%, +400 basis points (cc), mainly driven by higher net sales Operating income grew +44% (cc, +38% USD); net income up +37% (cc, +34% USD)Core EPS1 grew +31% (cc, +27% USD) to USD 2.28Free cash flow1 of USD 3.4 billion (+66% USD) driven by higher net cash flows from operating activities Selected innovation milestones: Pluvicto FDA approval for pre-taxane mCRPC Vanrafia (atrasentan) FDA accelerated approval for IgA nephropathy Fabhalta (iptacopan) FDA, EC and China NMPA approvals for C3G Remibrutinib global submissions for CSU, with priority review voucher in US OAV101 IT Phase III STEER study positive readout in SMA Full-year 2025 guidance2 raised: Sales expected to grow high single digit, core operating income expected to grow low double-digit Basel, April 29, 2025 – Commenting on Q1 2025 results, Vas Narasimhan, CEO of Novartis, said:“Novartis has had a strong start to the year, delivering a +15% cc increase in sales and a +27% cc rise in core operating income in Q1. Our priority brands, including Kisqali, Kesimpta and Leqvio, continue to show strong momentum, which we anticipate will drive our growth through 2030 and beyond. We also achieved significant innovation milestones in the quarter, with new approvals for Pluvicto in the pre-taxane setting, Vanrafia for IgA nephropathy, and Fabhalta for C3G. Additionally, we completed global submissions for remibrutinib in CSU, the first indication for this promising pipeline-in-a-pill. We remain focused on advancing our leading pipeline and confident in achieving our growth outlook.” Key figures Q1 2025Q1 2024% change USD m USD mUSDccNet sales to third parties13 23311 8291215Operating income4 6633 3733844Net income3 6092 6883437EPS (USD)1.831.314042Free cash flow3 3912 03866 Core operating income5 5754 5372327Core net income4 4823 6812226Core EPS (USD)2.281.802731 1. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 31 of the Condensed Interim Financial Report. Unless otherwise noted, all growth rates in this Release refer to same period in prior year. 2. Please see detailed guidance assumptions on page 5. Strategy Our focus Novartis is a “pure-play” innovative medicines company. We have a clear focus on four core therapeutic areas (cardiovascular-renal-metabolic, immunology, neuroscience and oncology), with multiple significant in-market and pipeline assets in each of these areas, that address high disease burden and have substantial growth potential. In addition to two established technology platforms (chemistry and biotherapeutics), three emerging platforms (gene & cell therapy, radioligand therapy and xRNA) are being prioritized for continued investment into new R&D capabilities and manufacturing scale. Geographically, we are focused on growing in our priority geographies – the US, China, Germany and Japan. Our priorities Accelerate growth: Renewed attention to deliver high-value medicines (NMEs) and focus on launch excellence, with a rich pipeline across our core therapeutic areas.Deliver returns: Continuing to embed operational excellence and deliver improved financials. Novartis remains disciplined and shareholder-focused in our approach to capital allocation, with substantial cash generation and a strong capital structure supporting continued flexibility. Strengthen foundations: Unleashing the power of our people, scaling data science and technology and continuing to build trust with society. Financials Net sales were USD 13.2 billion (+12%, +15% cc), with volume contributing 15 percentage points to growth. Generic competition had a negative impact of 2 percentage points and pricing had a positive impact of 2 percentage points, benefiting from revenue deduction adjustments mainly in the US. Operating income was USD 4.7 billion (+38%, +44% cc), mainly driven by higher net sales, partly offset by higher investments behind priority brands and launches. Net income was USD 3.6 billion (+34%, +37% cc), mainly driven by higher operating income, partly offset by higher income taxes. EPS was USD 1.83 (+40%, +42% cc), benefiting from the lower weighted average number of shares outstanding. Core operating income was USD 5.6 billion (+23%, +27% cc), mainly driven by higher net sales, partly offset by higher investments behind priority brands and launches, and R&D investments. Core operating income margin was 42.1% of net sales, increasing 3.7 percentage points (4.0 percentage points cc). Core net income was USD 4.5 billion (+22%, +26% cc), mainly due to higher core operating income. Core EPS was USD 2.28 (+27%, +31% cc), benefiting from the lower weighted average number of shares outstanding. Free cash flow amounted to USD 3.4 billion (+66% USD), compared with USD 2.0 billion in the prior-year quarter, driven by higher net cash flows from operating activities. Q1 priority brands Underpinning our financial results in the quarter is a continued focus on key growth drivers (ranked in order of contribution to Q1 growth) including: Entresto(USD 2 261 million, +22% cc) sustained robust, demand-led growth globally, including in China and Japan with increased penetration in hypertensionKisqali(USD 956 million, +56% cc) sales grew strongly across all regions, including +87% growth in the US with strong momentum from the recently launched early breast cancer indication as well as continued share gains in metastatic breast cancerKesimpta(USD 899 million, +43% cc) sales grew across all regions driven by increased demand and strong accessCosentyx(USD 1 534 million, +18% cc) sales grew mainly in the US, emerging growth markets and Europe, driven by recent launches as well as volume growth in core indicationsLeqvio(USD 257 million, +72% cc) continued steady growth, with a focus on increasing account and patient adoption, and continuing medical educationScemblix(USD 238 million, +76% cc) sales grew across all regions, demonstrating the continued high unmet need in CML and strong momentum from the recently launched early-line indication in the USFabhalta(USD 81 million) sales grew driven by continued launch execution across all markets in PNH as well as the recent launch in IgAN in the USPluvicto(USD 371 million, +21% cc) continued stable performance in the US and grew in Europe in the mCRPC post-taxane setting. With the FDA’s approval for earlier use before chemotherapy, which approximately triples the eligible patient population, the focus is on driving demand in established RLT sites while activating new sites and supporting referring providers to enable patient accessZolgensma(USD 327 million, +13% cc) sales grew as it continues to demonstrate strong performance in the incident populationLutathera(USD 193 million, +15% cc) sales grew mainly in the US, Europe and Japan due to increased demand and earlier line adoption particularly in the US and Japan Net sales of the top 20 brands in the first quarter Q1 2025 % change USD mUSDccEntresto2 2612022Cosentyx1 5341618Kisqali 9565256Kesimpta 8994143Tafinlar + Mekinist 5521619Promacta/Revolade 54658Jakavi 49237Xolair 4561419Ilaris 4191820Tasigna 377-5-2Pluvicto 3712021Zolgensma 3271113Sandostatin Group 317-11-9Leqvio 2577072Scemblix 2387576Lutathera 1931415Lucentis 189-40-38Exforge Group 179-7-1Diovan Group 150712Galvus Group 124-17-11Top 20 brands total10 8371719 R&D update - key developments from the first quarter New approvals Pluvicto(lutetium Lu177 vipivotide tetraxetan)FDA expanded the indication for Pluvicto to include patients with PSMA-positive metastatic castration-resistant prostate cancer (mCRPC) who have been treated with an androgen receptor pathway inhibitor and are considered appropriate to delay chemotherapy, approximately tripling the eligible patient population. Vanrafia(atrasentan)FDA granted an accelerated approval for Vanrafia for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. Vanrafia can be seamlessly added to supportive care in IgAN and used as a foundational therapy.Fabhalta(iptacopan)Fabhalta was approved by the US FDA, European Commission and China’s NMPA for adult patients with C3 glomerulopathy (C3G), making it the first and only treatment approved for this condition in all three markets. Regulatory updates RemibrutinibRegulatory submissions for remibrutinib for the treatment of chronic spontaneous urticaria (CSU) were completed in the US, EU and China. A priority review voucher was used in the US and approval is anticipated in H2 2025, and priority review was granted in China. Scemblix(asciminib)Regulatory submission for Scemblix in adults with newly diagnosed CML was completed in the EU based on 96-week data from the Phase III ASC4FIRST study. Results from ongoing trials and other highlights OAV101 IT(onasemnogene abeparvovec)Novartis announced positive safety and efficacy results from the Phase III program for investigational intrathecal OAV101 IT in a broad population of patients aged two to <18 years with spinal muscular atrophy (SMA). In the Phase III STEER study, treatment with OAV101 IT led to a statistically significant and clinically meaningful 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded vs. 0.51 points in the sham control arm. In addition, in the Phase IIIb STRENGTH study, treatment with OAV101 IT in patients who have discontinued treatment with nusinersen or risdiplam demonstrated stabilization of motor function over 52 weeks of follow-up. OAV101 IT demonstrated a favorable safety profile, consistent in both treatment-naïve and treatment-experienced patients. Data were presented at MDA.RemibrutinibThe New England Journal of Medicine published data from the 24-week double-blind placebo-controlled period of the Phase III REMIX-1 and -2 studies. Remibrutinib showed early symptom improvement and sustained efficacy, with improvements in CSU symptoms compared to placebo observed as early as week 1 and response rate maintained through the double-blind treatment period. Remibrutinib was well tolerated, with overall adverse event rates comparable to placebo. Multiple analyses of the REMIX-1 and -2 trials were presented at medical congresses in Q1. At AAAAI, long-term results showed that patients experienced improved urticaria control within two weeks of starting treatment. Patients who switched from placebo to remibrutinib at week 24 achieved similar improvements through week 52. At AAD, data was presented showing that treatment with remibrutinib had positive effects on sleep and daily activities for CSU patients.IanalumabThe results of Phase II VAYHIT3 study in adult patients with advanced primary ITP, previously treated with at least a corticosteroid and a thrombopoietin receptor agonist, indicate that a short course of ianalumab has clinically meaningful efficacy and is well tolerated in these patients. These results will be presented at a future medical meeting and are expected to support a filing in second-line ITP, based on the Phase III VAYHIT2 study, expected to read out in H2 2025.Selected transactionsNovartis has completed the acquisition of Anthos Therapeutics, a clinical-stage biopharmaceutical company developing abelacimab, a potential first-in-class monoclonal antibody targeting the FXI inhibition pathway in development for the prevention of stroke and systemic embolism in patients with atrial fibrillation. The acquisition adds a Phase III asset and is aligned with the Novartis growth strategy and expertise in the cardiovascular therapeutic area. Capital structure and net debt Retaining a good balance between investment in the business, a strong capital structure, and attractive shareholder returns remains a priority. In Q1 2025, Novartis repurchased a total of 24.8 million shares for USD 2.6 billion on the SIX Swiss Exchange second trading line under the up-to USD 15 billion share buyback announced in July 2023 (with up to USD 2.7 billion still to be executed). In addition, 1.5 million shares (equity value of USD 0.2 billion) were repurchased from employees. In the same period, 10.5 million shares (equity value of USD 0.3 billion) were delivered to employees related to equity-based compensation plans. Novartis aims to offset the dilutive impact from equity-based compensation plans of employees over the remainder of the year. Consequently, the total number of shares outstanding decreased by 15.8 million versus December 31, 2024. These treasury share transactions resulted in an equity decrease of USD 2.5 billion and a net cash outflow of USD 2.7 billion. Net debt increased to USD 22.3 billion at March 31, 2025, compared with USD 16.1 billion at December 31, 2024. The increase was mainly due to the free cash flow of USD 3.4 billion being more than offset by the USD 5.3 billion annual net dividend payment in March (which is the gross dividend of USD 7.8 billion reduced by the USD 2.5 billion Swiss withholding tax that was paid in April 2025, according to its due date), cash outflows for treasury share transactions of USD 2.7 billion and cash outflows for purchases of intangible assets of USD 1.2 billion. As of Q1 2025, the long-term credit rating for the company is Aa3 with Moody’s Ratings and AA- with S&P Global Ratings. 2025 outlook Barring unforeseen events; growth vs. prior year in ccNet salesExpected to grow high single-digitCore operating incomeExpected to grow low double-digit Key assumptions: We assume Tasigna, Promacta and Entresto US generic entry mid-2025 for forecasting purposes Foreign exchange impact If late-April exchange rates prevail for the remainder of 2025, the foreign exchange impact for the year would be 0 percentage points on net sales and negative 2 percentage points on core operating income. The estimated impact of exchange rates on our results is provided monthly on our website. Key figures1 Q1 2025Q1 2024% change USD m USD mUSDccNet sales to third parties13 23311 8291215Operating income4 6633 3733844As a % of sales35.228.5 Net income3 6092 6883437EPS (USD)1.831.314042Net cash flows from operating activities3 6452 26561 Non-IFRS measures Free cash flow 3 3912 03866 Core operating income5 5754 5372327As a % of sales42.138.4 Core net income4 4823 6812226Core EPS (USD)2.281.802731 1. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 31 of the Condensed Interim Financial Report. Unless otherwise noted, all growth rates in this Release refer to same period in prior year. Detailed financial results accompanying this press release are included in the Condensed Interim Financial Report at the link below:https://ml-eu.globenewswire.com/resource/download/f5843473-7c9e-4c13-b263-db739fdcf6d6/ DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995, that can generally be identified by words such as “anticipate,” “can,” “will,” “continue,” “ongoing,” “growth,” “launch,” “expect,” “expand,” “deliver,” “accelerate,” “deliver,” “guidance,” “outlook,” “priority,” “potential,” “momentum,” or similar expressions, or by express or implied discussions regarding potential new products, potential new indications for existing products, potential product launches, or regarding potential future revenues from any such products; or regarding results of ongoing clinical trials; or regarding potential future, pending or announced transactions; regarding potential future sales or earnings; or by discussions of strategy, plans, expectations or intentions, including discussions regarding our continued investment into new R&D capabilities and manufacturing; or regarding our capital structure. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. You should not place undue reliance on these statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. Neither can there be any guarantee that the expected benefits or synergies from the transactions described in this press release will be achieved in the expected timeframe, or at all. In particular, our expectations could be affected by, among other things: uncertainties concerning global healthcare cost containment, including ongoing government, payer and general public pricing and reimbursement pressures and requirements for increased pricing transparency; uncertainties regarding the success of key products, commercial priorities and strategy; uncertainties in the research and development of new products, including clinical trial results and additional analysis of existing clinical data; our ability to obtain or maintain proprietary intellectual property protection, including the ultimate extent of the impact on Novartis of the loss of patent protection and exclusivity on key products; uncertainties regarding our ability to realize the strategic benefits, operational efficiencies or opportunities expected from our external business opportunities; uncertainties in the development or adoption of potentially transformational digital technologies, including artificial intelligence, and business models; uncertainties surrounding the implementation of our new IT projects and systems; uncertainties regarding potential significant breaches of information security or disruptions of our information technology systems; uncertainties regarding actual or potential legal proceedings, including regulatory actions or delays or government regulation related to the products and pipeline products described in this press release; safety, quality, data integrity, or manufacturing issues; our performance on and ability to comply with environmental, social and governance measures and requirements; major macroeconomic and geo- and socio-political developments, including the impact of any potential tariffs on our products or the impact of war in certain parts of the world; uncertainties regarding future global exchange rates; uncertainties regarding future demand for our products; and other risks and factors referred to in Novartis AG’s most recently filed Form 20-F and in subsequent reports filed with, or furnished to, the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements as a result of new information, future events or otherwise. All product names appearing in italics are trademarks owned by or licensed to Novartis. About Novartis Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide. Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on LinkedIn, Facebook, X and Instagram. Novartis will conduct a conference call with investors to discuss this news release today at 14:00 Central European time and 8:00 Eastern Time. A simultaneous webcast of the call for investors and other interested parties may be accessed by visiting the Novartis website. A replay will be available after the live webcast by visiting https://www.novartis.com/investors/event-calendar. Detailed financial results accompanying this press release are included in the Condensed Interim Financial Report at the link below. Additional information is provided on our business and pipeline of selected compounds in late-stage development. A copy of today's earnings call presentation can be found at https://www.novartis.com/investors/event-calendar. Important datesJuly 17, 2025October 28, 2025November 19-20, 2025Second quarter & half year 2025 resultsThird quarter & nine months 2025 resultsMeet Novartis Management 2025 (London, UK) Novartis Media RelationsE-mail: media.relations@novartis.com Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com Please find full media release in English attached and on the following link: Media release (PDF) Further language versions are available through the following links: German version is available through the following link: Medienmitteilung (PDF)

Clinical ResultPhase 3Drug ApprovalPhase 2Acquisition

18 Apr 2025

·www.fiercepharma.com

Sanofi, Regeneron score their 7th indication for Dupixent with FDA nod to treat hives condition

The FDA has signed off on Sanofi and Regeneron’s megablockbuster Dupixent to treat patients with chronic spontaneous urticaria. It is the fourth new indication approval for Dupixent in the last 16 months and seventh overall. On its way to generating sales of $14 billion in just its seventh full year on the market, Sanofi and Regeneron’s Dupixent has experienced few setbacks. One came in 2023, however, when the FDA rejected it as a treatment for chronic spontaneous urticaria (CSU), asking for more data.Eighteen months later—and backed by more conclusive results—the companies have convinced the U.S. regulator to sign off on Dupixent for the difficult-to-treat skin condition. With the nod, Dupixent becomes the first new medicine for CSU in more than a decade. The approval clears Dupixent to be used by those age 12 and older who remain symptomatic despite using histamine-1 (H1) antihistamines. It’s a population of more than 300,000 among the roughly 3 million in the U.S. who have CSU, the companies said in a release.The incidence of the inflammatory skin disease is highest among women between the ages of 30 and 50. CSU causes sudden, unpredictable hives and a severe itch.“This FDA approval provides a new treatment option to help address the underlying drivers of these severe and recurring signs and symptoms. Dupixent has the potential to improve outcomes for CSU patients who previously had limited treatment options,” Alyssa Johnsen, M.D., Ph.D., Sanofi’s global lead for immunology and oncology development, said in a statement.Dupixent was first rejected for CSU after the failure of a phase 3 trial that enrolled 108 patients who did not have success with the only other treatment approved in the indication, Novartis and Roche’s Xolair. In the Liberty-Cupid B study, Dupixent failed to significantly improve symptoms, though numerical improvements were evident in each of the main evaluation criteria. The next trial of Dupixent in the indication, Liberty-Cupid C, achieved its primary and secondary objectives. In the study, which included 148 CSU patients who were already receiving standard-of-care (SOC) antihistamine therapy, adding Dupixent provided a reduction of 8.6 points in itch severity (on a scale of 21) compared to a 6.1-point reduction for patients on placebo plus SOC.The Dupixent group also saw a 15.9-point reduction in itch and hive severity (on a 42-point scale) versus an 11.2-point reduction for the control group. Additionally, 30% of Dupixent-treated patients experienced a complete response, compared with 18% of those on placebo. The measurements were taken after 24 weeks of therapy.The trial confirmed results from the successful Liberty-Cupid A study, which also tested Dupixent plus SOC antihistamines against antihistamines alone in 136 patients with moderate-to-severe CSU. CSU is the seventh disease with underlying type 2 inflammation in which Dupixent is approved. The others are atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps, eosinophilic esophagitis, prurigo nodularis and chronic obstructive pulmonary disease (COPD).Dupixent has been approved as a treatment for CSU in Japan, Brazil and the United Arab Emirates, and is under review for the indication in Europe.Xolair, which was approved for CSU in 2014 and covers some of the same indications as Dupixent, generated sales of $4.7 billion last year. With Xolair soon to face biosimilar competition, Novartis is working to bring a new CSU treatment to the market in BTK inhibitor remibrutinib. The oral treatment has succeeded in two phase 3 trials.

Phase 3Clinical ResultDrug Approval

09 Apr 2025

·www.echemi.com

Novartis Launches Bold Attack Eyes 100 Billion Dollar Autoimmune Market

Novartis has reignited its competitive streak, targeting the reign of Dupixent (dupilumab), the newly crowned “autoimmune drug king” from Sanofi. The company has initiated a head-to-head Phase III trial for its investigational BTK inhibitor remibrutinib against Dupixent, aiming to disrupt the dominance of this top-tier therapeutic. This is not Novartis’ first strike at market leaders. In the past, its blockbuster Cosentyx (secukinumab) outperformed AbbVie's Humira and Johnson & Johnson’s Stelara in direct head-to-head trials, cementing its position as a key player in psoriasis treatment. However, despite these wins, Novartis’ 2024 autoimmune business revenue of $9.293 billion left it short of the $10 billion milestone and outside the top three in the field. Industry giants such as AbbVie, Johnson & Johnson, and Sanofi dominate the autoimmune space with super blockbuster drugs. AbbVie leads with a commanding $11.7 billion in Skyrizi sales and strong growth from Rinvoq, while Johnson & Johnson relies on products like Stelara and Tremfya. Sanofi, with Dupixent achieving $14.2 billion in sales, has solidified its position as the third-largest player in the sector. Novartis, though just shy of the $10 billion mark, is not backing down. Cosentyx, its flagship IL-17A inhibitor, has driven significant revenue growth, reaching $6.141 billion in 2024 sales, thanks to approvals for multiple indications like plaque psoriasis and psoriatic arthritis. Expansion into conditions such as polymyalgia rheumatica (PMR) and giant cell arteritis (GCA), with Phase III trial results expected in 2025, could further elevate its market potential to $8 billion in peak sales. Yet, its other autoimmune drugs lag behind. Xolair (omalizumab), co-developed with Roche, generated $1.643 billion outside the US, but faces increasing pressure from Dupixent, which is now approved for chronic spontaneous urticaria (CSU) in Japan and awaiting FDA approval. Additionally, Chinese competitors are advancing innovative IgE-targeting therapies, challenging Xolair’s dominance. To counter these threats, Novartis has taken bold steps. It became the first company to file for remibrutinib’s approval in CSU treatment in China and has begun the RECLAIM Phase III trial, comparing remibrutinib directly with Dupixent. As a highly selective, oral, and irreversible BTK inhibitor, remibrutinib offers convenience over injectable biologics, with Novartis projecting $3 billion in peak sales. Additionally, Novartis secured an $830 million deal with Kyorin Pharmaceutical to develop MRGPRX2 antagonists like KRP-M223, targeting allergic and inflammatory mast cell diseases such as CSU. This emerging class of drugs represents a blue ocean market, with few competitors currently in clinical trials. Novartis is also advancing a robust autoimmune pipeline, with 15 early-stage and 8 late-stage clinical programs exploring diverse mechanisms, including NLRP3 inhibitors, CD19 CAR-T therapies, and BAFF-R monoclonal antibodies. Analysts suggest that Novartis’ strategy of head-to-head trials, pipeline diversification, and strategic licensing could help secure its place in the $100 billion autoimmune market. While challenges remain, including strong competition from Sanofi and Johnson & Johnson, Novartis’ aggressive moves could reshape the landscape, positioning it as a leading force in the autoimmune sector.

Phase 3Drug ApprovalLicense out/inImmunotherapy

100 Deals associated with Remibrutinib

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Chronic Urticaria	NDA/BLA	China	Beijing Novartis Pharma Co. Ltd.	27 Feb 2025
Chronic Urticaria	NDA/BLA	China	Novartis Pharma Schweiz AG	27 Feb 2025
Chronic Urticaria	NDA/BLA	China	Novartis Pharmaceutical Manufacturing GmbH	27 Feb 2025
Myasthenia Gravis	Phase 3	United States	Novartis Pharmaceuticals Canada, Inc.	07 Feb 2025
Hidradenitis Suppurativa	Phase 3	United States	Novartis Pharmaceuticals Canada, Inc.	31 Jan 2025
Hidradenitis Suppurativa	Phase 3	United States	China Novartis Institutes for BioMedical Research Co., Ltd.	31 Jan 2025
Hidradenitis Suppurativa	Phase 3	China	China Novartis Institutes for BioMedical Research Co., Ltd.	31 Jan 2025
Hidradenitis Suppurativa	Phase 3	Argentina	Novartis Pharmaceuticals Canada, Inc.	31 Jan 2025
Hidradenitis Suppurativa	Phase 3	Argentina	China Novartis Institutes for BioMedical Research Co., Ltd.	31 Jan 2025
Hidradenitis Suppurativa	Phase 3	Australia	China Novartis Institutes for BioMedical Research Co., Ltd.	31 Jan 2025

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05795153 (CTgov)	Phase 3	Chronic Urticaria	144	LOU064	bughlvruxw(puokuaynml) = tphwcmxomx vwopvxcsoa (ixkpnfuexd, mhbrvemuwb - rprhuzzzpj) View more	-	30 Apr 2025
NCT05032157 \| NCT05030311 (REMIX-2, NEWS \| Pubmed) Manual	Phase 3	Urticaria	-	remibrutinib (REMIX-1)	cxsmujomnr(svdfhefwvv) = kryoclowih dqrhepmdcp (krmxfzicgw, 0.7) View more	Positive	11 Mar 2025
				placebo (REMIX-1)	cxsmujomnr(svdfhefwvv) = mqbjpzpfww dqrhepmdcp (krmxfzicgw, 1.0) View more
REMIX-1 and REMIX-2 (Pubmed \| N Engl J Med)	Phase 3	Chronic Urticaria Bruton's tyrosine kinase	-	Remibrutinib 25 mg twice daily	dqymgpnatt(olncxemwqt) = tqaeppdlig elwihzdjvy (houpagmvyi ) View more	Positive	06 Mar 2025
				Placebo	dqymgpnatt(olncxemwqt) = roelkjkvea elwihzdjvy (houpagmvyi ) View more
NCT05030311 (REMIX-1, CTgov)	Phase 3	Chronic Urticaria	470	LOU064 (LOU064 25mg b.i.d.)	fccoijqbmb(qdgcphwtjh) = kiulbnvqhs ptipjckyco (obwqutcrej, 0.716) View more	-	02 Dec 2024
				Placebo+LOU064 (Placebo)	fccoijqbmb(qdgcphwtjh) = qffepzarbl ptipjckyco (obwqutcrej, 0.980) View more
NCT05048342 (BISCUIT, CTgov)	Phase 3	Chronic Urticaria	71	LOU064	mtndmvwona = kqxqiolxed bljrbuizvr (wopojxbaub, atlfzeewgi - xuymgzzmgr) View more	-	22 Nov 2024
NCT05032157 (REMIX-2, CTgov)	Phase 3	Chronic Urticaria	455	LOU064 (open-label)+Placebo	mnunlvozdq(mieuqexeee) = iilxyrgkox dgomwodjud (pumybxcvpp, 0.948) View more	-	01 Nov 2024
REMIX-1/-2 (ACAAI2024)	Phase 3	Chronic Urticaria Bruton's tyrosine kinase (BTK)	-	Remibrutinib 25 mg twice daily	jwismkzbea(mbfoxzqpgu) = vivtgnzkoy faygxxlmut (ypybcvxbbn ) View more	Positive	24 Oct 2024
NCT03926611 (Phase 2b core and extension study, EAN2024)	Phase 2	Serum Sickness	309	Remibrutinib 100 mg b.i.d.	drrqckomht(qkvfsfpxly) = xpnahzxlvu xjlgiibwiv (ciasybjgrf, 2.47) View more	Positive	28 Jun 2024
NCT05030311 (REMIX-1, GlobeNewswire) Manual	Phase 3	Chronic Urticaria	470	Remibrutinib	wpdnhvumbp(chzlpatdjd) = Almost half of patients were completely free of itch and hives (UAS7=0) as assessed at Week 52 mfwxggtdcz (bankqlrylg ) View more	Positive	30 May 2024
				Placebo
NCT05032157 (REMIX-2, GlobeNewswire) Manual	Phase 3	Chronic Urticaria	455	Remibrutinib	sruchjakoz(ckpveetyjk) = Almost half of patients were completely free of itch and hives (UAS7=0) as assessed at Week 52 axzqenokkf (gdjrsuhrbg ) View more	Positive	30 May 2024
				Placebo

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